Gene Therapy's Second Act

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A decade and a half after a series of tragic setbacks led to critical reevaluations, scientists say gene therapy is ready to enter the clinic

Gene therapy may finally be living up to its early promise. In the past six years the experimental procedure for placing healthy genes wherever they are needed in the body has restored sight in about 40 people with a hereditary form of blindness. Doctors have seen unprecedented results among another 120-plus patients with various cancers of the bloodseveral of whom remain free of malignancy three years after treatment. Researchers have also used gene therapy to enable a few men with hemophilia, a sometimes fatal bleeding disorder, to go longer without dangerous incidents or the need for high doses of clotting drugs.

The positive results are even more impressive considering that the field of gene therapy essentially ground to a halt 15 years ago, following the untimely death of Jesse Gelsinger, a teenager with a rare digestive disorder. Gelsinger's immune system reacted to the gene treatment he received by launching a counterattack of unexpected ferocity that killed him. Gene therapy's preliminary successes in the 1990s, it turns out, had fueled unreasonably high expectations among doctors and researchersand perhaps a bit of hubris.

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Gene Therapy's Second Act

Chemist Direct discusses Gene therapy that offers hope to thousands of people suffering from degenerative blindness

London (PRWEB UK) 3 March 2014

Researchers at Oxford University have discovered that by replacing a missing gene into the retina, they can prevent cells from degenerating. Scientists hope that early intervention with the surgical treatment will halt progression of the devastating disorder before patients are entirely robbed of their sight. (http://bit.ly/1gOIohJ)

Superintendent Pharmacist at ChemistDirect, Omar El-Gohary, said: A lot of degenerative eye diseases result from faulty genes. Gene therapy is a new development, which uses technology to replace the defective genes with a normal working copy, with a single injection.

It is the first time gene therapy has successfully been applied to the light-sensitive photoreceptors of the retina, the digital camera at the back of the eye.

Results from the preliminary results done by Oxford University researchers has shown that from the first six patients to take part, two men with advanced stages of choroideremia, (a degenerative retinal disease which leads to loss in sight) experienced dramatic improvements to their sight.

A third of diseases which affect the eyes are genetic in origin and scientists are confident the therapy could be adapted to help patients with other illnesses.

El-Gohary added: Factors other than genetics contributing to the development of eye related diseases can be mutations, which can occur with age and trigger macular disease. The treatment is the same; to replace the faulty gene with a functioning one.

There are currently 500,000 people in Britain with age-related degenerative macular disease, with one in 20 people over 65 suffering from the disease. This pioneering technology could make an enormous difference to the lives of thousands. (http://bit.ly/1fhvH1I)

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Chemist Direct discusses Gene therapy that offers hope to thousands of people suffering from degenerative blindness

FDA mulling 3-parent embryos

By Matt Smith, CNN

updated 7:06 PM EST, Thu February 27, 2014

STORY HIGHLIGHTS

(CNN) -- A promising way to stop a deadly disease, or an uncomfortable step toward what one leading ethicist called eugenics?

U.S. health officials are weighing whether to approve trials of a pioneering in vitro fertilization technique using DNA from three people in an attempt to prevent illnesses like muscular dystrophy and respiratory problems. The proposed treatment would allow a woman to have a baby without passing on diseases of the mitochondria, the "powerhouses" that drive cells.

The procedure is "not without its risks, but it's treating a disease," medical ethicist Art Caplan told CNN's "New Day" on Wednesday. Preventing a disease that can be passed down for generations would be ethical "as long as it proves to be safe," he said.

"These little embryos, these are people born with a disease, they can't make power. You're giving them a new battery. That's a therapy. I think that's a humane ethical thing to do," said Caplan, the director of medical ethics at New York University's Langone Medical Center.

"Where we get into the sticky part is, what if you get past transplanting batteries and start to say, 'While we're at it, why don't we make you taller, stronger, faster or smarter?' "

But Susan Solomon, the director of the New York Stem Cell Foundation, said there are no changes to existing genes involved.

"There is no genetic engineering. It isn't a slippery slope. It's a way to allow these families to have healthy children," said Solomon, whose organization developed the technique along with Columbia University researchers.

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FDA mulling 3-parent embryos

'Cyborg' has Rousey in her sights

Ronda Rousey secured a TKO victory against Sara McMann in 66 seconds Getty Images

Invicta FC featherweight champion Cristiane Justino has a plan for 2014, which she hopes will lead to UFC bantamweight champion Ronda Rousey by December.

Justino (12-1) recently hired medical consultants to help her start shedding weight in an attempt to make the 135-pound bantamweight limit by midsummer.

The plan calls for Justino, aka 'Cyborg', to defend her 145-pound Invicta title in late April or early May, most likely against Ediane Gomes. Justino would then fight for Invicta's 135-pound title in a quick turnaround, perhaps as early as July.

In previous interviews, the Brazilian fighter hinted she was entering the final fight on the Invicta contract she signed last year. Invicta president Shannon Knapp wouldn't offer details, but said the proposed two title fights wouldn't necessarily require Justino to sign a contract extension.

"I won't confirm or deny where she is on her contract, but I can tell you she would still be contractually under Invicta on her current deal [in that situation]," Knapp told ESPN.

A former Strikeforce champion, Justino publicly stated her intent to drop to 135 pounds last Saturday, hours before Rousey (9-0) was scheduled to defend her title against Sara McMann at UFC 170 in Las Vegas. Rousey won the fight via first-round TKO.

Justino is scheduled to compete in a 145-pound muay Thai bout at Lion Fight 14 on March 28 in Las Vegas. According to her attorney George Prajin, Justino typically weighs approximately 170 pounds between fights and has a hard time even cutting to her current weight class.

The goal is for Justino to gradually drop, under a physician's supervision, over the next few months, which would make her next two cuts to 145 pounds easier. She would then make a practice cut to 135 before fighting for the Invicta bantamweight title this summer.

Earlier this month, Justino's manager, former UFC champion Tito Ortiz, stepped down from his involvement with her. UFC president Dana White has criticised Ortiz's handling of Justino's career in the past.

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'Cyborg' has Rousey in her sights

Ronda Rousey Rumored to Fight Gina Carano or Cris Cyborg in July

Isaac Brekken/Associated Press

The first ever womens MMA superfight could be on the horizon, pitting UFC champ Ronda Rousey against either Cristiane Cyborg Justino or Gina Carano.

During an appearance on KROQs Kevin & Bean Show on Friday (h/t MMAFighting.com), UFC commentator Joe Rogan teased at a huge announcement coming from the UFC, possibly within the next week, regarding Rouseys next opponent.

Within the next probably week or so a huge announcement will come about women's fighting and I'll be back in [the studio] and we'll talk some more, Rogan said. It's going to be crazy. Madness. I wish I could [talk about it now], but I would betray the confidence of my friend and employer.

UFC President Dana White recently told Yahoo! Sports Kevin Iole that Rousey was the biggest star in UFC history.

Unfortunately, pay-per-view numbers arent generated by compliments. Rouseys headliner against fellow Olympian Sara McMann a little more than a week ago at UFC 170 didnt live up to Whites forecast in buys at the pre-fight media scrum. This was especially disappointing considering McMann was penned as Rouseys biggest fight left in the womens bantamweight division.

It also didnt help that it took Rousey only 66 seconds to dispatch of McMann and record her third straight UFC title defense.

An appetite for marquee fights seems insatiable at this point, considering the glaring gap in skill between Rousey and the rest of the bantamweight division. Fortunately for fans, hope could lie in Rogans teaser of a major announcement forthcoming from the UFC.

MMA journalist Dave Meltzer echoed Rogans comments in the Wrestling Observer (h/t BloodyElbow.com), and he named Carano and Cyborg, two of MMAs biggest stars, as the two potential opponents:

Rousey has two potential opponents, both of which would draw huge for different reasons. The first is Gina Carano. Carano hasn't fought in four-and-a-half years. There would be hell to pay in criticism for UFC if they were to make that fight, if Carano would even have interest in it.

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Ronda Rousey Rumored to Fight Gina Carano or Cris Cyborg in July

Biologists Hope For Another Busy Sea Turtle Nesting Season [GALLERY]

News Release: Florida Fish and Wildlife Conservation Commission

More sea turtles nest on Floridas sandy beaches than on any other U.S. coastline. Biologists at the Florida Fish and Wildlife Conservation Commission (FWC) hope the 2014 nesting season that started March 1 will be as successful as others in recent years.

This month, leatherback sea turtles begin to emerge onto beaches to lay their eggs along Floridas Atlantic coast, from Broward to Brevard counties. A few months later, people in other coastal counties also may notice loggerhead and green sea turtle crawls, the distinctive line of tracks they leave behind in the sand.

Three species of sea turtles nest in abundance on Florida beaches: leatherbacks, loggerheads and greens. Loggerheads are the most abundant, and approximately 90 percent of all nests for this species in the southeastern United States occur in Florida. Sea turtle biologists were surprised and pleased in 2013 when a record number of more than 36,000 green sea turtle nests were counted in Florida.

The great news is that so many sea turtles nest on Florida beaches more than anywhere else in the United States, said Dr. Robbin Trindell, who is responsible for sea turtle management at the FWC. Florida had a record number of loggerhead nests in 2012, followed by a record number of green turtle nests in 2013.

Typically, sea turtle nesting season runs from March through the end of October, but nesting continued well beyond that in 2013. Green turtles generally nest later than the other sea turtle species in Florida. FWC biologists would not be surprised if nesting season extends later into the fall again this year, and they caution beachgoers that marked nests on the beach may hatch well past the official end of nesting season in October. The actions that people take are critical to maintaining Floridas success with sea turtles, Trindell said. Remove chairs, canopies, boats and other items from the beach at night, because they block the movement of turtles and hatchings. Dont forget to turn off or shield lights on the beach, to prevent hatchlings from getting confused and going toward land instead of the salt water where they belong.

In Florida, sea turtle landings on the beach are documented by volunteers, who assist the FWCs researchers. About 2,500 FWC-permitted volunteers regularly patrol more than 800 miles of sandy shoreline to identify, mark and protect sea turtle nests. They collect nesting data and also share their knowledge with beachgoers on how to help conserve sea turtles.

Support Floridas sea turtles by purchasing the Helping Sea Turtles Survive license tag at BuyaPlate.com. Tag funds are spent on sea turtle research, rescue and conservation efforts. People also can donate $5 and receive an FWC sea turtle decal. Learn more about sea turtles at MyFWC.com/SeaTurtle.

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Biologists Hope For Another Busy Sea Turtle Nesting Season [GALLERY]